Clinical trials: Deficiency Disease
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood…
- Trial name
- NCT06090669
- Testing
- TruSight Oncology, imatinib
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
HIV Infections
PrEP Seguro: Antiretroviral-based HIV Prevention Among Men at High Risk in Mexico
The objective of this R34 application is to prepare for testing innovative user-centered ways to promote PrEP adherence at scale.
- Trial name
- NCT03674983
- Testing
- Conditional Economic Incentive (CEI)
- Where
- Mexico
- Volunteers
- 310 people, healthy volunteers welcome
- Sponsor
- Brown University
Infection, Human Immunodeficiency Virus
Dose-finding Study of GSK2248761 in Antiretroviral Therapy-experienced Subjects With NNRTI-resistant HIV Infection
This 48 week, phase 2b study in 150 HIV-1 infected antiretroviral therapy experienced adult subjects consists of a dose-ranging evaluation of GSK2248761 at blinded doses of 100 mg and 200 mg once daily with a control arm of…
- Trial name
- SONNET (NCT01199731)
- Testing
- GSK2248761 100 mg once daily, GSK2248761 200 mg once daily, Etravirine
- Where
- 41 sites in United States, Belgium and 3 more
- Volunteers
- 30 people
- Sponsor
- ViiV Healthcare
HIV Infections
An Open-Label Study to Evaluate the Efficacy and Safety of Amprenavir (141W94) and Abacavir Combination Therapy in Protease Inhibitor Experienced Subjects With HIV-1 Infection Who Are Failing Their Current Antiretroviral Treatment Regimen
To assess the safety, tolerance, and efficacy of amprenavir (APV) plus abacavir (ABC) in patients who have previously failed antiretroviral treatment containing a protease inhibitor (PI).
- Trial name
- NCT00002205
- Testing
- Abacavir sulfate, Amprenavir
- Where
- United States
- Sponsor
- Glaxo Wellcome
HIV/AIDS
Study in Healthy Males to Measure Maraviroc in Blood, Saliva, Seminal Fluid, and Rectal Tissue
This study is being conducted to look at how the body handles the drug maraviroc. It will measure the amount of maraviroc in blood, semen, saliva and in the rectum of men.
- Trial name
- NCT00775294
- Testing
- Maraviroc
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Severe Combined Immunodeficiency
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)
Infants with severe combined immunodeficiency (SCID) have a profound decrease in number and function of immune cells, and therefore remain highly vulnerable to infection. If not corrected this often leads to death.
- Trial name
- NCT03597594
- Testing
- Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
HIV Infections
Study of the Antiviral and Immunological Effects of Intensification of Suppressive Antiretroviral Therapy With Maraviroc
Hypothesis: Intensification of current ARV regimens with maraviroc will result in more complete suppression of viral replication, particularly in the gastrointestinal mucosa with resultant reduction in markers of immune…
- Trial name
- NCT00703586
- Testing
- Maraviroc
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Rockefeller University
Alpha1-Antitrypsin Deficiency
An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein.
- Trial name
- NCT05899673
- Testing
- Fazirsiran Injection
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 31 people
- Sponsor
- Takeda
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Hepatitis B, HIV Infection
Adefovir Dipivoxil to Treat Hepatitis B in HIV-Infected Patients
This study will evaluate the safety and effectiveness of adding the experimental drug adefovir dipivoxil to lamivudine for treating hepatitis B virus (HBV) infection in HIV-infected patients with liver cirrhosis.
- Trial name
- NCT00013702
- Testing
- Adefovir
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Anemia
Safety and Tolerability of Ferric Carboxymaltose (FCM) Versus Standard of Care in Treating Iron Deficiency Anemia
The objective of this study is to evaluate the safety of FCM in patients with anemia who are not dialysis dependent.
- Trial name
- NCT00703937
- Testing
- Ferric Carboxymaltose (FCM), Standard Medical Care (SMC) for the treatment of IDA
- Where
- United States
- Volunteers
- 708 people
- Sponsor
- American Regent, Inc.
HIV Infections
Boosted Atazanavir and Truvada Given Once-Daily - BATON Study
To determine the safety and efficacy of a simple, once-daily antiretroviral (ARV) regimen consisting of a fixed-dose combination tablet containing Truvada combined with atazanavir boosted with ritonavir in treatment naive…
- Trial name
- NCT00224445
- Testing
- Truvada, Atazanavir, Ritonavir
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Gilead Sciences
Campylobacter Infections
Recurrent Campylobacter Bacteraemia in Immunocompromised Patients
Some rare cases of recurrent Campylobacter bacteraemia (RCB) exist with relapses months to years after an effective treatment and a negativation of all bacterial samples.
- Trial name
- CABARET (NCT06432777)
- Testing
- Characteristics of patients and Campylobacter bacteraemia episodes
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
Albinism, Inborn Errors of Metabolism
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Hermansky-Pudlak Syndrome (HPS) is an inherited disease that results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal…
- Conditions
- Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency
- Trial name
- NCT00001596
- Testing
- Pirfenidone
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- William Gahl, M.D.
Primary Hyperoxaluria, Dent Disease
Rare Kidney Stone Consortium Patient Registry
The purpose of this study is to collect medical information from a large number of patients in many areas of the world with primary hyperoxaluria (PH), Dent disease, Cystinuria and APRT deficiency.
- Conditions
- Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
- Trial name
- RKSC (NCT00588562)
- Where
- 4 sites in United States, Iceland
- Volunteers
- 730 people
- Sponsor
- Mayo Clinic
HIV INFECTIONS
Immunization With HIV-1 Peptides in Adjuvant for Treatment of Patients With Chronic HIV-infection
Treatment: Immunization with a peptide-mix of 17 Clusters of Differentiation number 8 (CD8) T cell minimal epitopes and 3 Clusters of Differentiation number 4 (CD4) T cell epitopes and a new adjuvant (CAF01).
- Trial name
- HIV-VAC (NCT01009762)
- Testing
- peptide vaccine (AFO-18)
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Gitte Kronborg
Human Immunodeficiency Virus Type 1
A Study of TMC278 in Human Immunodeficiency Virus Type 1 Infected Patients, Who Are Not Treated With Antiretroviral Medicines
The purpose of this study is to evaluate the dose-response relationship of antiviral activity after 48 weeks treatment with 3 different dose regimens of TMC278.
- Trial name
- NCT00110305
- Testing
- TMC278 25 mg, TMC278 75 mg, TMC278 150 mg
- Where
- 40 sites in United States, Argentina and 12 more
- Volunteers
- 368 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
Anemia, HIV Infections
An Open-Label Study to Evaluate the Effect of Every Other Week PROCRIT� (Epoetin Alfa) Dosing (40,000-60,000 Units) On Maintaining Quality of Life and Target Hemoglobin Levels in Anemic HIV-Infected Patients (CHAMPS II)
The objective of this study was to treat anemic (Hemoglobin (Hb) < 12 g/dL) HIV-infected subjects with once weekly (QW) PROCRIT (Epoetin alfa) to a target Hb of > 13 g/dL and then to assess if the target Hb level and improvements…
- Trial name
- NCT00317902
- Testing
- Epoetin alfa
- Volunteers
- 292 people
- Sponsor
- Ortho Biotech Products, L.P.
HIV Infections
Drug-drug Interaction (DDI) Study of GSK3640254 With Darunavir/Ritonavir (DRV/RTV) and Etravirine (ETR)
This is an open-label, single-sequence, multiple-dose, 3 cohort study to investigate the effects of DRV/RTV and/or ETR on the pharmacokinetics (PK) of GSK3640254 and the effects of GSK3640254 on the PK of DRV/RTV and/or ETR.
- Trial name
- NCT04630002
- Testing
- GSK3640254, Darunavir/Ritonavir (DRV/RTV), Etravirine (ETR)
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Growth Hormone Deficiency
Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects…
- Trial name
- OraGrowtH210 (NCT04614337)
- Testing
- LUM-201, rhGH Norditropin® pen (34 µg/kg)
- Where
- 43 sites in United States, Australia and 4 more
- Volunteers
- 104 people
- Sponsor
- Lumos Pharma
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